Patient-reported Outcomes for Clinical Trials in Idiopathic Pulmonary Fibrosis: New Opportunities to Understand How Patients Feel and Function

Holland, Anne E; Dowman, Leona; Smallwood, Natasha

doi:10.1164/rccm.202007-2775ed

Cited by 2 publications

(2 citation statements)

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“…Most systematic data reflecting the use of PROMs in IPF or f-ILD have been derived from interventional studies [ 9 , 10 ]. PROMs have served primarily as secondary outcomes in randomised controlled trials.…”

Section: Discussionmentioning

confidence: 99%

“…Patient-reported outcome measures (PROMs) are proctored or self-administered questionnaires that directly elicit the impressions or insights of patients regarding their symptom burden or HRQoL [9][10][11] . Prior studies in interstitial lung disease (ILD) have reported mixed association of PROMs with presenting lung function or radiologic findings 1,12 Most interventional studies still include PROMs as secondary or adjunctive outcomes as often required by local governing or oversight bodies 7,11,13 .…”

Section: Introductionmentioning

confidence: 99%

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Association of patient-reported outcome measures with lung function and mortality in fibrotic interstitial lung disease: a prospective cohort study

Duke,

Roy,

Daley

et al. 2024

ERJ Open Res

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BACKGROUNDPatient-reported outcome measures (PROMs) may provide clinicians and researchers with direct insights into disease impact and patient well-being. We assessed whether selected PROMs and their domains are associated with baseline and longitudinal changes in lung function and can predict mortality in patients with fibrotic interstitial lung disease (f-ILD).METHODSA single-center prospective study of adult patients with f-ILD enrolled over three years was conducted assessing baseline and short-term changes in PROMs. Three questionnaires, the modified Medical Research Council dyspnea scale (mMRC), Chronic Respiratory Questionnaire (CRQ), and Self-Management Ability Scale (SMAS-30) were administered at planned intervals and assessed for their association with baseline clinical findings, change in lung function (percent predicted forced vital capacity (FVC%) and diffusion capacity for carbon monoxide (DLCO%)), and all-cause mortality.RESULTS199 patients were enrolled with a mean PROM follow-up of 9.6 months. When stratified by FVC% quartiles at presentation, lower mMRC (less dyspnea), higher CRQ Physical and Emotional domain (better HRQoL) and higher total SMAS-30 scores (better self-management ability) were associated with higher FVC%. Short-term changes in all three PROMs appeared to be associated with changes in FVC% and DLCO%. Adjusted and unadjusted baseline and serial PROM changes were also predictive of mortality.CONCLUSIONSBaseline and serial assessments of PROMs were associated with changes in lung function and predicted death in patients with f-ILD. PROMs may strengthen comprehensive assessments of disease impact in clinical practice as well as support patient-centered outcomes in research.

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Section: Discussionmentioning

confidence: 99%